On March 26, 2014, the FDA hosted the Fibromyalgia Public Meeting on Patient-Focused Drug Development. The meeting was moderated by Sara Eggers, PhD, Operations Research Analyst at the Office of Program and Strategic Analysis of the FDA’s Center for Drug Evaluation and Research. Fibromyalgia (FM) is one of the 20 disease areas targeted by the FDA to consider patients’ needs in drug development. At the FM meeting, the FDA invited people living with the disease to share their symptoms, treatment regimens and side effects. They also discussed needs that have been unmet by currently available treatments and offered suggestions for what the FDA and healthcare industry should focus on.
During the meeting, many people living with FM expressed that they’ve encountered a vicious cycle between lack of sleep and other FM symptoms such as fatigue, pain, cognitive impairment and gastrointestinal issues. Some people living with FM said that although current treatments were effective at managing pain or mood, the side effects of those therapies can be daunting and some have caused problems with sleeping. Due to various side effects and mechanisms, many people living with FM discussed a plethora of therapies that they’ve tried either separately or in combination with others. The consensus is current treatment options are generally unsatisfactory.
When asked to describe the major symptoms that people living with FM experienced, one patient said, “I’m so exhausted, yet I can’t sleep.” When asked what people living with FM would like in a therapy, another patient responded, “If I could have a perfect treatment, it would regulate my sleep cycle.”
Practicing and teaching rheumatology for nine years at Columbia University, where he became a tenured professor, Seth Lederman, MD heard many similar complaints from his patients and recognized that they are desperate for new options. As CEO of Tonix Pharmaceuticals, Inc., he, along with the rest of the Tonix team, is striving to address this treatment challenge.
Tonix is a development-stage pharmaceutical company developing innovative prescription medications to treat FM, post-traumatic stress disorder and episodic tension-type headache, all characterized by inadequate treatment options, dissatisfaction expressed among patients and physicians, and significant expense burden. The company’s investigation therapy, TNX-102 SL, is a sublingual formulation of cyclobenzaprine (which is an FDA-approved muscle relaxant at higher doses for oral ingestion) currently being studied in a national clinical study called BESTFIT. TNX-102 SL, which comes in an easy-to-take sublingual form, is designed to improve FM by targeting their disturbed sleep, recognized to have negative impact on FM symptoms. Additionally, the sublingual form is designed to decrease next-day side effects such as grogginess due to how it is absorbed by the body.
Dr. Lederman is available to discuss:
• What are the most common symptoms of FM as described by patients?
• Why have large pharmaceutical companies failed to satisfactorily address the need for better treatments for FM?
• What is the link between lack of sleep and other FM symptoms?
• Why might addressing sleep quality be the key to new and effective FM treatments?
• What are the advantages of reformulating an existing drug for use as an FM treatment rather than starting from scratch?
At a time when the concerns of FM patients with their symptoms and current treatments are becoming more widely known, Dr. Lederman and Tonix are in the forefront of researchers who are searching for innovative new alternatives.